Liver fibrosis negatively impacts in vivo gene transfer to murine hepatocytes.

ABSTRACT: Liver fibrosis occurs in several genetic and acquired disease conditions, leading to alterations of the tissue and metabolism, which may adversely affect viral vector-mediated gene therapy. Here, we assessed the impact of liver fibrosis on in vivo gene transfer to hepatocytes mediated by lentiviral vectors or adeno-associated viral vectors. We exploited two chemically induced fibrosis mouse models characterized by tissue damage in different areas of the liver lobule. Moreover, we used Abcb11^-/- and Agl^-/- mice, recapitulating features of inherited cholestasis and glycogen storage disease, as representative models of genetic disorders characterized by liver fibrosis. We report a general negative influence of liver fibrosis on hepatocyte transduction and alte

SUBMITTER: Simoni C 

PROVIDER: S-EPMC11894088 | biostudies-literature | 2025 Mar

REPOSITORIES: biostudies-literature